Active not recruitingPHASE2INTERVENTIONAL

Azacitidine With or Without Lenalidomide or Vorinostat in Treating Patients With Higher-Risk Myelodysplastic Syndromes or Chronic Myelomonocytic Leukemia

This research study is for adults diagnosed with specific types of blood conditions, like higher-risk myelodysplastic syndromes (MDS) or chronic myelomonocytic leukemia (CMML). Doctors are testing a treatment called azacitidine, which is a common chemotherapy drug. They want to find out if adding another drug, either lenalidomide or vorinostat, makes azacitidine more effective. Lenalidomide aims to stop cancer growth by reducing blood flow to the cells, while vorinostat tries to block enzymes needed for cell growth. This study is trying to discover the best combination to help patients live longer and improve their condition. It's a randomised trial, meaning participants will be assigned to a treatment group by chance.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

National Cancer Institute (NCI)

Enrolment target

282

Start

01 Mar 2012

Estimated completion

31 Mar 2027

Chronic Myelomonocytic Leukemia Chronic Myelomonocytic Leukemia-1 Chronic Myelomonocytic Leukemia-2 Myelodysplastic Syndrome Myelodysplastic Syndrome With Excess Blasts Myelodysplastic Syndrome With Excess Blasts-1 Myelodysplastic Syndrome/Acute Myeloid Leukemia

Results

Results from this study

Posted March 2016

Results have been published for this study.

Primary outcome

Response Rate (Phase II)

A response is any of complete hematological remission, partial remission, or hematologic improvement.

Full results on the registry

What is this study about?

This research study is for adults who have been diagnosed with certain types of blood conditions. These include myelodysplastic syndromes (MDS) that are considered 'higher-risk' or a condition called chronic myelomonocytic leukemia (CMML). These conditions can affect how your body makes healthy blood cells.

The main aim of this study is to see if a normal chemotherapy drug, called azacitidine, works better when combined with one of two other drugs: lenalidomide or vorinostat. Azacitidine helps to stop cancer cells from growing and spreading. Lenalidomide is thought to work by cutting off the blood supply to cancer cells, which can stop them from growing. Vorinostat tries to block certain chemicals (enzymes) that cancer cells need to grow. By combining these treatments, doctors hope to find the most effective way to manage these conditions and improve patients' health.

This study is being done in two main parts, called phases. In the first part (Phase II), the doctors will compare the azacitidine combinations to see which one looks most promising. In the second part (Phase III), they will take the best combination and compare it against azacitidine on its own, to see if the combined treatment helps patients live longer. The hope is that these combinations might offer a better treatment option for people with these challenging blood disorders.

Key takeaways

Tests if combining azacitidine with other drugs improves treatment.
For adults with higher-risk MDS or CMML.
Compares azacitidine alone to azacitidine plus lenalidomide or vorinostat.
Participation involves regular clinic visits for treatment and checks.
Helps doctors find better ways to treat these blood conditions.

Who may be eligible?

To join this study, you must be an adult aged 18 or over. You need to have a confirmed diagnosis of certain types of myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML. Specifically, if you have MDS, it needs to be of a 'higher-risk' type, and if you have CMML, it also needs to meet certain criteria related to the number of young blood cells (blasts) in your bone marrow or blood.

There are also some important points about your past treatments. You cannot have received lenalidomide, azacitidine, vorinostat, or another drug called decitabine before. If you've taken certain growth factors or other medications for your condition, you'll need to have stopped them for a specific amount of time before joining the study. For example, if you've had low-dose cytarabine, you must have stopped it at least 28 days before, and for hydroxyurea, at least 7 days before. People with acute myeloid leukemia (AML) are not able to take part in this specific trial.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years old or older?
Have you been diagnosed with higher-risk MDS or CMML?
Have you *not* taken azacitidine, lenalidomide, vorinostat, or decitabine before?
Are you able to stop any other specific blood medications for a period before starting?
Do you *not* have acute myeloid leukemia (AML)?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will be assigned by chance to one of two or three different treatment groups, as this is a 'randomised' study. This means neither you nor your doctor will get to choose which group you are in. Each group will receive a specific combination of drugs: either azacitidine alone, or azacitidine with lenalidomide, or azacitidine with vorinostat.

The azacitidine will be given either into a vein (intravenously) or under the skin (subcutaneously) on specific days during each treatment cycle. If you receive lenalidomide, it will be a tablet you take by mouth once a day. If you receive vorinostat, it will also be a tablet taken by mouth. You will have regular visits to the clinic for your treatments, blood tests, and other assessments to see how you are responding and if you are experiencing any side effects. The study will look at how your condition changes, your general health, and your quality of life. The exact total duration of your participation will depend on how you respond to treatment and will be explained fully by the study team.

Potential risks and benefits

Taking part in a clinical trial has potential benefits and risks. You might benefit from receiving a new combination of treatments that could be more effective than standard care for your condition, though this is not guaranteed. Your participation also helps doctors learn more about these conditions and how to treat them, which could help future patients. However, there are potential risks, including side effects from the medications, which might be new or more severe than known side effects. The study team will monitor you closely for any unwanted effects. You are always free to withdraw from the study at any time, for any reason, without it affecting your future medical care.

Locations (294)

University of Alabama at Birmingham Cancer Center
Verified postcode
Birmingham, United States
University of South Alabama Mitchell Cancer Institute
Verified postcode
Mobile, United States
Mayo Clinic in Arizona
Verified postcode
Scottsdale, United States
Banner University Medical Center - Tucson
Verified postcode
Tucson, United States
University of Arizona Cancer Center-North Campus
Verified postcode
Tucson, United States
John L McClellan Memorial Veterans Hospital
Verified postcode
Little Rock, United States
Kaiser Permanente-Deer Valley Medical Center
Verified postcode
Antioch, United States
City of Hope Comprehensive Cancer Center
Verified postcode
Duarte, United States
USC / Norris Comprehensive Cancer Center
Verified postcode
Los Angeles, United States
Kaiser Permanente-Oakland
Verified postcode
Oakland, United States
Stanford Cancer Institute Palo Alto
Verified postcode
Palo Alto, United States
Kaiser Permanente-Richmond
Verified postcode
Richmond, United States

Common questions

What is 'higher-risk' MDS or CMML?

These are types of blood conditions where your body doesn't make enough healthy blood cells. 'Higher-risk' means they are more likely to get worse over time compared to lower-risk forms.

What drugs are being tested?

The main drug is azacitidine, a chemotherapy. Researchers are testing if adding lenalidomide or vorinostat to azacitidine makes the treatment more effective.

Will I choose which treatment I get?

No, because it's a 'randomised' study, participants are assigned to a treatment group by chance, like flipping a coin. This helps make the study fair.

How long will the treatment last?

The duration of treatment will vary for each person, depending on how your body responds to the medication and whether you experience side effects. The study team will explain this in detail.

Can I stop participating if I want to?

Yes, you are free to leave the study at any time, for any reason, and it will not affect your usual medical care.